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How transplant can treat MLD

A bone marrow or cord blood transplant is the only known treatment that can stabilize the neurologic effects of metachromatic leukodystrophy (MLD). The transplant replaces the patient’s blood-forming cells missing the important enzyme with healthy ones from the donor that contain the enzyme.

The type of transplant used for MLD is an allogeneic transplant. This type of transplant uses healthy blood-forming cells from a family member, unrelated donor, or umbilical cord blood unit.

For an allogeneic transplant, a patient gets chemotherapy, with or without radiation, prior to transplant to prepare his or her body for the treatment. Then, the replacement cells are infused into the patient’s blood stream. From there, the cells find their way into the bone marrow, where they start making healthy white blood cells, red blood cells and platelets. These new cells provide the body with the ability to break down the fat-based compound built up and can stop further damage.

The entire process, from the start of chemotherapy or radiation until hospital discharge, can last weeks to months followed by many months of recovery at home.

Understanding if transplant would help your child with MLD

Because children with MLD have physical and mental problems that get worse over time, it is best to have a transplant as soon as possible after diagnosis. Children who get a transplant early enough, before a lot of damage has happened, can have normal or near-normal mental and physical development.

Transplants for children with MLD who have already developed severe damage have had poor results. Therefore it’s important to move forward with transplant as quickly as possible after diagnosis. For parents who know that MLD runs in the family, children can be tested at birth and transplant can be planned to get the best possible results. If the disease was not known in the family before, it may take some time to diagnose. Moving forward with transplant as soon as possible helps to minimize permanent damage.

For these reasons, doctors recommend that children with MLD be referred to a transplant doctor as soon as they are diagnosed.1 A transplant doctor who is an expert in MLD can explain the risks and benefits of transplant.

1Recommended Timing for Transplant Consultation. Guidelines developed jointly by National Marrow Donor Program/Be The Match and the American Society for Blood and Marrow Transplantation (ASBMT). Available at: marrow.org/md-guidelines